� Regeneron Pharmaceuticals, Inc. (Nasdaq: REGN) announced the publication of the results of trey studies which supported the U.S. Food and Drug Administration (FDA) regulatory meekness for ARCALYST� (rilonacept) Injection for Subcutaneous Use for the treatment of Cryopyrin-Associated Periodic Syndromes (CAPS).
Two studies were published in the August 2008 outcome of Arthritis and Rheumatism, the flagship publication of the American College of Rheumatology. The first study was the preliminary trial to measure the shock of ARCALYST on markers of excitement, clinical response, and safety and the second was the pivotal efficacy and safety clinical trial of ARCALYST. A third sketch, conducted to develop and validate a key symptoms assessment scale for CAPS patients and subsequently utilised as the primary endpoint measure in the polar clinical subject of ARCALYST, was published online in the August 2008 issue of Current Medical Research and Opinion.
ARCALYST, besides known as interleukin-1 (IL-1) Trap, is a targeted inhibitor of IL-1, which is the key driver of inflaming in CAPS. ARCALYST is the merely therapy sanctioned for patients with CAPS, including Familial Cold Auto-inflammatory Syndrome (FCAS) and Muckle-Wells Syndrome (MWS) in adults and children 12 and older. CAPS represent a group of rare, inherited, auto-inflammatory weather condition characterized by life-long, repeated symptoms of rash, fever/chills, joint hurting, eye redness/pain, and fatigue. Intermittent, tumultuous exacerbations or flares lav be triggered at whatsoever time by exposure to cooling temperatures, stress, exercise, or other unknown stimuli. The relative incidence of CAPS has been reported to be around one in 1,000,000 citizenry in the United States.
"The ultimate goal of our exploit over the last ten years has been to find an effective therapy for Cryopyrin-Associated Periodic Syndromes. We began by characterizing the unusual clinical features and debilitating impact of CAPS and then discovered the inherent genetic fundament of the disease. A major concern was whether a pharmaceutic company would expend the significant resources necessary to develop a treatment for a disease that has been diagnosed in only a few hundred patients in the United States," stated Hal Hoffman, M.D., Associate Professor, University of California, San Diego and a preeminent expert on CAPS. "These publications highlight that scientists, clinicians, and industry tin collaborate to efficiently, still rigorously, develop therapies for rare diseases with meaning unmet medical need. As a consequence of this collaboration, patients with CAPS now receive an approved treatment available that backside help them to effectively manage their disease."
In the initial, open-label airplane pilot evaluation of ARCALYST� (rilonacept), five patients with the Familial Cold Auto-inflammatory Syndrome (FCAS) sub-type of CAPS experienced a marked improvement in symptoms with a corresponding simplification in markers of inflaming. Based upon these convincing findings, a pivotal clinical program was designed to support a registration filing. In ordering to develop a ground for mensuration symptom rigorousness as reported by CAPS patients in a disease that waxes and wanes from twenty-four hours to day, an observational, non-pharmacologic cogitation was conducted in 48 patients with either FCAS or Muckle-Wells Syndrome (MWS). Systematic evaluation of a series of patient-reported outcomes tools resulted in the development of a validated CAPS symptoms measurement official document with high internal consistence and dependability.
In the pivotal, randomized, double-blind clinical study, 47 patients with FCAS or MWS were randomized to receive either ARCALYST or placebo for six weeks. Patients treated with ARCALYST experienced an 84 percentage statistically significant improvement in overall symptom scores versus baseline, whereas those treated with placebo did non experience a significant improvement in their symptoms (13 percent improvement). Ninety-six percent of patients receiving ARCALYST experienced at least a 30 percentage improvement in symptoms during this six-week phase of the study. All patients were later given single-blind ARCALYST for nine weeks. Patients were then re-randomized to either ARCALYST or placebo and evaluated over a subsequent nine-week period. Patients odd on ARCALYST continued to experience symptom control, whereas those receiving placebo experient a statistically significant worsening of their CAPS symptoms. The nigh commonly reported adverse reactions reported with ARCALYST were injection-site reaction and upper berth respiratory parcel infection.
"The development serve for ARCALYST for the treatment of CAPS highlights Regeneron's boilersuit approach to drug maturation - to develop drugs targeted at well-documented mediators of disease, focus clinical development on diseases in which those biologic mediators play a primary implicit in role, set up clinical proof of concept, and then strive to conduct efficient, pivotal studies with well-validated clinical endpoints," stated George D. Yancopoulos, M.D., Ph.D., President of Regeneron Research Laboratories. "In this type, recognizing that IL-1 is an active mediator of inflammatory disease, we developed ARCALYST to potently inhibit IL-1 in the bloodstream before it can bond to its receptors. Once it was recognized that the genetical mutation associated with CAPS is associated with IL-1 overproduction, we rapidly initiated a pilot study to determine the clinical impact of IL-1 inhibition with ARCALYST. Based upon the clear-cut responses experienced by the CAPS patients in the pilot study, we then developed a validated instrument to assess the severity of CAPS symptoms over metre and introduced that cat's-paw into deuce randomized, placebo-controlled pivotal study phases."
About Cryopyrin-Associated Periodic Syndromes (CAPS)
Recently, aesculapian researchers have identified and described a group of rare, hereditary, auto-inflammatory disorders, known as Cryopyrin-Associated Periodic Syndromes or CAPS. Three related conditions make up the broader disease known as CAPS: Familial Cold Auto-inflammatory Syndrome (FCAS), Muckle-Wells Syndrome (MWS), and Neonatal-Onset Multisystem Inflammatory Disease (NOMID). ARCALYST has not been studied in patients with NOMID.
CAPS are characterized by womb-to-tomb, recurrent symptoms of rash, fever/chills, joint pain, eye redness/pain, and fatigue. Intermittent, disruptive exacerbations or flares can be triggered at any time by photo to chilling temperatures, tenseness, exercise, or other unknown stimuli.
CAPS are generally caused by autosomal-dominant mutations (changes) in the NLRP-3 (previously known as CIAS1) gene and resultant alterations in the protein, cryopyrin, which it encodes. Cryopyrin, active in circulating infection-fighting white descent cells, controls the production of a protein called interleukin-1 (IL-1). As part of the body's infection-fighting defense system, IL-1 circulates throughout the body and can trigger inflammatory reactions when it binds to inflammatory cells. Researchers have found that alterations in the cryopyrin protein pencil lead to overrun of IL-1, resulting in an inflammatory response and the symptoms of CAPS. Most, just not all, patients with CAPS have the NLRP-3 gene sport.
Important Information About ARCALYST� (rilonacept)
ARCALYST is indicated for the treatment of Cryopyrin-Associated Periodic Syndromes (CAPS), including Familial Cold Auto-inflammatory Syndrome (FCAS) and Muckle-Wells Syndrome (MWS) in adults and children 12 and older. IL-1 blockade may interfere with immune response to infections. Serious, dangerous infections give been reported in patients taking ARCALYST. ARCALYST should be discontinued if a patient ps a serious infection. Taking ARCALYST with tumor necrosis factor inhibitors is not recommended because this may addition the peril of grave infections. Treatment with ARCALYST should non be initiated in patients with active or chronic infections. Patients should non receive a live vaccinum while pickings ARCALYST. It is recommended that patients receive all recommended vaccinations prior to initiation of treatment with ARCALYST. Patients should be monitored for changes in their lipid profiles and provided with medical treatment if warranted. Hypersensitivity reactions associated with ARCALYST organization have been rare. Please see the full Prescribing Information for ARCALYST, available online at http://www.regeneron.com/ARCALYST-fpi.pdf.
About Regeneron Pharmaceuticals, Inc.
Regeneron is a fully unified biopharmaceutical society that discovers, develops, and commercializes medicines for the treatment of serious aesculapian conditions. In addition to ARCALYST� (rilonacept) Injection for Subcutaneous Use, its first-class honours degree commercialized product, Regeneron has therapeutic candidates in clinical trials for the potential treatment of cancer, eye diseases, and inflammatory diseases, and has preclinical programs in other diseases and disorders. Additional information around Regeneron and recent newsworthiness releases are available on Regeneron's web site at http://www.regeneron.com.
Forward Looking Statement
This news program release discusses historical information and includes forward-looking statements about Regeneron and its products, exploitation programs, monetary resource, and clientele, all of which affect a number of risks and uncertainties, such as risks associated with preclinical and clinical development of Regeneron's drug candidates, determinations by regulative and administrative governmental government which may delay or restrict Regeneron's ability to continue to develop or commercialize its product and drug candidates, competing drugs that ar superior to Regeneron's ware and dose candidates, precariousness of market acceptance of Regeneron's product and drug candidates, unlooked-for expenses, the availability and cost of capital, the costs of developing, producing, and marketing products, the potential for any collaborationism agreement, including Regeneron's agreements with the sanofi-aventis Group and Bayer HealthCare, to be canceled or to terminate without any product success, risks associated with third party intellectual property, and other material risks. A more than complete description of these and other material risks can be found in Regeneron's filings with the United States Securities and Exchange Commission (SEC), including its Form 10-K for the year ended December 31, 2007 and Form 10-Q for the quarter ending June 30, 2008. Regeneron does not guarantee any obligation to update publicly whatsoever forward-looking statement, whether as a event of new information, future events, or otherwise unless required by law.
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